Safety and pharmacokinetics of ONC201 (dordaviprone) administered two consecutive days per week in pediatric patients with H3 K27M-mutant glioma.

doi:10.1093/neuonc/noae001

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Article

Safety and pharmacokinetics of ONC201 (dordaviprone) administered two consecutive days per week in pediatric patients with H3 K27M-mutant glioma.

Neuro-oncology - 3 May 2024

Odia Yazmin, Koschmann Carl, Vitanza Nicholas A, de Blank Peter, Aguilera Dolly, Allen Jeffrey, Daghistani Doured, Hall Matthew, Khatib Ziad, Kline Cassie, MacDonald Tobey, Mueller Sabine, Faison Shamia L, Allen Joshua E, Naderer Odin J, Ramage Samuel C, Tarapore Rohinton S, McGovern Susan Lynne, Khatua Soumen, Zaky Wafik, Gardner Sharon L

Abstract excerpt

BACKGROUND: This study evaluated the safety and pharmacokinetics (PK) of oral ONC201 administered twice-weekly on consecutive days (D1D2) in pediatric patients with newly diagnosed DIPG and/or recurrent/refractory H3 K27M glioma. METHODS: This phase 1 dose-escalation and expansion study included pediatric patients with H3 K27M-mutant glioma and/or DIPG following ≥1 line of therapy (NCT03416530). ONC201 was...

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Identifiers

PMID: 38400780
DOI: 10.1093/neuonc/noae001
PMCID: PMC11066921

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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